Advances in pediatric rhabdomyosarcoma characterization and disease model development

Rhabdomyosarcoma (RMS), a form of soft tissue sarcoma, is one of the most common pediatric malignancies. A complex disease with at least three different subtypes, it is characterized by perturbations in a number of signaling pathways and genetic abnormalities. Extensive clinical studies have helped classify these tumors into high and low risk groups to facilitate different treatment regimens. Research into the etiology of the disease has helped uncover numerous potential therapeutic intervention points which can be tested on various animal models of RMS; both genetically modified models and tumor Xenograft models. Taken together, there has been a marked increase in the survival rate of RMS patients but the highly invasive, metastatic forms of the disease continue to baffle researchers. This review aims to highlight and summarize some of the most important developments in characterization and in vivo model generation for RMS research, in the last few decades

A Standards based Ontological Approach to Information Handling for use by Organizations Providing Human Tissue for Research

Tissue resources have become an important component of the infrastructure of institutions as well as companies performing biomedical research. Such tissue resources may be in the model of a bank, collecting a limited type of tissues and processing and storing them following a specifi c protocol. Such banks or archives may be associated with a clinical study or may function indepedently. An alternative type of tissue resource is utilized by many institutions and cancer centers. In this model, the investigator specifies the methods by which selected tissues are to be collected, processed and stored. In such a “prospective model”, initially developed at the University of Alabama at Birmingham and the Ohio State University in the late 1970’s and adopted by the Cooperative Human Tissue Network in 1986, specific types of tissues are not collected unless requested by an investigator. At some sites, both a prospective and an archival (bank) model are followed. This article describes an informatics approach needed to support a prospective tissue resource. It is by necessity more complicated than a model which supports a tissue bank but also can be used by a tissue bank. Of great importance is the approach to vocabulary and common data elements needed to support the informatics system of a prospective tissue resource

Results of treatment of fifty-six patients with localized retroperitoneal and pelvic rhabdomyosarcoma: a report from The Intergroup Rhabdomyosarcoma Study-IV, 1991-1997

BACKGROUND: We reviewed 56 IRS-IV patients with localized rhabdomyosarcoma [RMS] of the retroperitoneum/pelvis to assess outcome and prognostic factors, including the value of initially excising \u3e or=50% of the tumor (debulking) before chemotherapy. METHODS: Patients had embryonal RMS [N=38], alveolar RMS [N = 7], RMS not otherwise specified [NOS, N = 7], or undifferentiated sarcoma [N = 4]. Fifteen patients were debulked; 41 patients were biopsied. All received VAC; most received radiotherapy. RESULTS: Estimated 5-year failure-free survival [FFS] and overall survival rates were 70 and 75%, respectively. FFS rates were better for patients /undifferentiated sarcoma. After adjusting for age and histological differences, FFS was better for patients whose tumor was debulked prior to beginning therapy [P = 0.02]. CONCLUSIONS: These results are superior to those of previous protocols for patients with RMS of the retroperitoneum/pelvis. Initial excision of \u3e or=50% of the tumor may be associated with increased FFS

The Intergroup Rhabdomyosarcoma Study Group (IRSG): Major Lessons From the IRS-I Through IRS-IV Studies as Background for the Current IRS-V Treatment Protocols

Purpose. To enumerate lessons from studying 4292 patients with rhabdomyosarcoma (RMS) in the Intergroup Rhabdomyosarcoma Study Group (IRSG, 1972–1997)